Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD

a study on Macular Degeneration Copy Number Variation Neovascular AMD Choroidal Neovascularization

Summary

Eligibility
for people ages 50-89 (full criteria)
Location
at UC Irvine UCLA UCSF
Dates
study started
completion around
Principal Investigator
by Robert Bhisitkul (ucsf)
Headshot of Robert Bhisitkul
Robert Bhisitkul

Description

Summary

ABBV-RGX-314 (also known as RGX-314) is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD or nAMD). Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-vascular endothelial growth factor (anti-VEGF) therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to maintain or prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every 4 to 16 weeks in frequency, to maintain efficacy. Due to the burden of these treatments, patients often experience a decline in vision with reduced frequency of treatment over time.

Official Title

A Randomized, Partially Masked, Controlled, Phase 2b/3 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 Gene Therapy in Participants With nAMD (ATMOSPHERE)

Details

This randomized, partially masked, active-controlled, Phase 2b/3 clinical study will evaluate the efficacy and safety of ABBV-RGX-314 gene therapy in participants with nAMD. The study will evaluate 2 dose levels of ABBV-RGX-314 relative to an active comparator. The primary endpoint of this study is the mean change from baseline in best-corrected visual acuity (BCVA) of ABBV-RGX-314 relative to ranibizumab at Week 54. Approximately 540 participants who meet the inclusion/exclusion criteria, will be enrolled into one of 3 arms.

Keywords

AMD, nAMD, Wet Age-related Macular Degeneration, wAMD, Wet AMD, CNV, Neovascular AMD, Neovascular Age-related Macular Degeneration, Choroidal Neovascularization, Age-Related Macular Degeneration, Macular Degeneration, Wet Macular Degeneration, Retinal Degeneration, Retinal Diseases, Eye Diseases, Ranibizumab, Angiogenesis Inhibitors, Angiogenesis Modulating Agents, Growth Substances, Physiological Effects of Drugs, Growth Inhibitors, Antineoplastic Agents, Gene therapy, Anti-vascular endothelial grown factory therapy, Anti-VEGF therapy, Pathologic Neovascularization, ABBV-RGX-314, Ranibizumab (LUCENTIS®)

Eligibility

You can join if…

Open to people ages 50-89

  1. Age ≥ 50 years and ≤ 89 years
  2. An ETDRS BCVA letter score between ≤ 78 and ≥ 40 in the study eye
  3. Diagnosis of subfoveal CNV secondary to AMD in the study eye previously treated with anti-VEGF
  4. Must be pseudophakic (at least 12 weeks postcataract surgery) in the study eye.
  5. Willing and able to provide written, signed informed consent for this study
  6. Participants must have demonstrated a meaningful response to anti-VEGF therapy at study entry

You CAN'T join if...

  1. CNV or macular edema in the study eye secondary to any causes other than AMD
  2. Subfoveal fibrosis or atrophy in the study eye, as determined by CRC
  3. Any condition in the investigator's opinion that could limit VA improvement in the study eye
  4. Active or history of retinal detachment, or current retinal tear that cannot be treated, in the study eye
  5. Advanced glaucoma or history of secondary glaucoma in the study eye
  6. History of intraocular surgery in the study eye within 12 weeks prior to randomization
  7. History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to Screening Visit 1
  8. Prior treatment with gene therapy
  9. Recent myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months

Locations

  • 193-University of California Irvine - School of Medicine - The Gavin Herbert Eye Institute withdrawn
    Irvine California 92697 United States
  • UCLA Doheny Eye Center accepting new patients
    Pasadena California 91105 United States
  • 137-UCSF Medical Center - Retina and Vitreous Clinic accepting new patients
    San Francisco California 94158 United States
  • 190-West Coast Retina Medical Group - San Francisco Office accepting new patients
    San Francisco California 94109-5520 United States
  • 133-Retina-Vitreous Associates Medical Group accepting new patients
    Beverly Hills California 90211 United States
  • 160-The Retina Partners - Encino accepting new patients
    Encino California 91436-2018 United States
  • 182-Orange County Retina Medical Group accepting new patients
    Santa Ana California 92705-3592 United States
  • 284- Salehi Retina Institute accepting new patients
    Huntington Beach California 92647-3601 United States
  • 121-Retina Consultants San Diego accepting new patients
    Poway California 92064-2526 United States
  • 187-Doheny Eye Institute - Doheny Eye Center accepting new patients
    Los Angeles California 90033-1035 United States

Lead Scientist at University of California Health

  • Robert Bhisitkul (ucsf)
    Professor, Ophthalmology, School of Medicine. Authored (or co-authored) 62 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
AbbVie
ID
NCT04704921
Phase
Phase 2/3 research study
Study Type
Interventional
Participants
Expecting 540 study participants
Last Updated